Concept paper: Improving Access to Orphan Drugs (11th ERTC Workshop)

 

It has been acknowledged over recent years that, while the EU Orphan Drugs Regulation 141/2000 has stimulated research and development of orphan medicinal products in the EU, equitable and timely access to approved Orphan Drugs for rare diseases patients remains an issue. As underlined by the final conclusions and recommendations on Pricing & Reimbursement of the EU High Level Pharmaceutical Forum, “Effective market access and utilisation vary strongly between and within Member States”.

To address this issue, several policy documents have recently called for an increased cooperation between EU level authorities and Member States in order to improve access to Orphan Drugs for people living with rare diseases.

 
 
A voz das pessoas com doenças raras na EuropaEURORDIS A voz internacional das pessoas com doenças raras, Rare Diseases InternacionalRare Disease International Reúne doentes, famílias e especialistas para partilhar experiências num fórum multilinguístico. RareConnect O programa Rare Barometer é uma iniciativa da EURORDIS para a realização de inquéritos para transformar a experiência das pessoas com doenças raras em números e factos que podem ser partilhados com os responsáveis pela tomada de decisões.Rare Barometer An international awareness raising campaign taking place on the last day of February each year, Rare Disease Day is a EURORDIS initiativeRare Disease Day Adira ao maior encontro europeu das partes interessadas no âmbito das doenças raras na Conferência Bienal Europeia sobre Doenças Raras e Produtos Órfãos. A ECRD é uma iniciativa da EURORDISEuropean Conference on Rare Diseases